Approximately one out of every 2,500 people in the world suffers from a deficiency of the Alpha-1 Antitrypsin or AAT protein. Untreated, this genetic disorder – which affects the liver and lungs – is always fatal. Now an Israeli bio-pharmaceutical company has developed a more efficient treatment delivery system, holding out the promise of both an improvement in a patient’s quality of treatment, and their quality of life.

Kamada is no newcomer to drug development and manufacturing. Founded in 1990, the company went public in 2005 and has more than 15 products on the market. But it is hoping that the next generation of its flagship product – an inhalable version of its AAT replacement treatment – will be its biggest winner yet.

VP of business development Yaron Cherny, tells ISRAEL21c just what’s so revolutionary about Kamada’s latest medication. AAT deficiency, he says, starts in the liver and results in chronic inflammation of the lungs, which slowly suffocates the patient. There are currently only two ways to treat the ailment: Via a lung and liver transplant or a lifetime of receiving AAT intravenously.

For most patients, where transplants are not an option, intravenous AAT delivery requires weekly hospitalization and medical support; it is also often prohibitively expensive – upwards of $100,000 a year. Insurance companies, which nearly always cover the treatment, are acutely aware of this fact.

Inhaling your meds

An inhalable version of AAT can be used at home, obviating a trip to the clinic. That both makes the process less expensive and grants the patient significantly more freedom. From a medical point of view, since the inflammation is in the lungs, inhaling the drug directly makes more therapeutic sense, not to mention that it avoids the risks of infection that comes with using an IV.

While there are three other firms with AAT intravenous solutions, Kamada is the only one with an inhalable version (competitor Talecris Biotherapeutics was working on one but dropped out earlier this year).

Still, people suffering from AAT deficiency shouldn’t rush to their doctors to demand the drug just yet. The inhalable product is now in Phase II and III tests in Europe, which are slated to be completed in 2012. Indeed, Kamada only received FDA approval for the intravenous version in July of this year. And, as Cherny explains, until a drug is officially approved, “there’s always uncertainty.”

The good news is that Kamada’s inhalable AAT has already succeeded in five clinical trials “with no adverse effect,” according to Cherny, who adds that “the safety is almost perfect.”

An additional benefit is that Kamada’s IV version is in liquid rather than powder form. That means it doesn’t need mixing before delivery, as do the medications from the other companies. “We don’t have to develop the drug all over again,” Cherny says, since “the liquid particles are the right size to go into the inhalation device.”

Good news for investors

Inhalable AAT has been approved for ‘orphan’ drug status, a form of marketing protection that is given to drugs for diseases affecting small numbers of people. As the developer of an orphan drug, Kamada has the exclusive rights to marketing inhalable AAT for 10 years in the US and 12 years in the European Union, which “for many companies, is better than a patent,” Cherny declares.

Yaron Cherny, VP business development at Kamada.

This should also put investors at their ease – it can take more than half a billion dollars and 10 years of development and testing to bring a biological drug to market.

Only 5,000 people in the US and 6,000 in Europe have been diagnosed with AAT deficiency. However, Cherny estimates there may be upwards of 200,000 people suffering from the genetic disorder in those two regions alone. That makes the potential market much more lucrative. And if inhalable AAT brings greater awareness to physicians, Kamada will have contributed to much more than its own corporate bottom line.

AAT replacement therapy may also have implications for people suffering from cystic fibrosis and bronchiectasis, Cherny reveals. And new research by Dr. Eli Lewis of Israel’s Ben Gurion University of the Negev suggests it may help those suffering from Type 1 diabetes, although this is not related to AAT deficiency and Kamada is not active in this area.

All of Kamada’s products are based on the company’s in-house developed chromatographic purification technology. This was originally used to create only the active ingredients in drugs, which large pharmaceutical companies would then mix together to create complete medications. Kamada now manufactures the entire drug for its products.

Deal with Baxter

In August, Kamada signed a strategic agreement with Baxter International for production and marketing of its intravenous AAT drug. Shipments will begin in September. It’s a deal that CEO David Tsur believes will help make the company profitable and could bring in hundreds of millions of dollars in revenues. Kamada will receive up to $45 million in milestone payments, including a $20m. upfront payment.

For inhalable AAT, the company has partnered with PARI, a German-based firm specializing in inhalation solutions.

In other sectors, Kamada has just registered its passive rabies vaccine in Russia, and expects to begin sales in the first half of 2011. The vaccine is derived from human plasma with additional antibodies, and is used to prevent rabies, which affects the central nervous system.

Kamada was founded by Argentinean Jewish immigrant Ralf Hahn, whose family has been active in the drug industry for years, and businessman Tzur.

The company, which is listed on the Tel Aviv Stock Exchange, has raised more than $139 million. Kamada’s stock price has increased more than 70 percent since 2007, despite the worldwide economic crisis.

Kamada has 250 employees, 30 in its Rishon Letzion headquarters, and 220 in the company’s manufacturing plant on Kibbutz Beit Kama. The name is a play on words composed of the name of the kibbutz plus the world mada, which is Hebrew for ‘science.’

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Brian has been a journalist and high-tech entrepreneur for over 20 years. He combines this expertise for ISRAEL21c as he writes about hot new local startups, pharmaceutical advances, scientific discoveries, culture, the arts and daily life in Israel. He loves hiking the country with his family (and blogging about it). Originally from California, he lives in Jerusalem with his wife and three children.